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The latest news from PTC

  1. 25 Remarkable Stories: Eat Without Fear

    Imagine being unable to experience the delight of eating food in a restaurant without fear. For people living with the rare disease familial chylomicronemia syndrome (FCS), this is their unfortunate reality. FCS, which affects one in 700,000 to one million people, prevents the body from breaking down fat, or triglycerides, leading to an accumulation in…
    Rare Disease Community
    June 7, 2023
    3 minutes
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  2. The Importance of Inclusive Leadership: 4 Questions About Leading EMEA with Jesse Sibarium

    Over the past 25 years, our mission to extend life’s moments for people living with rare diseases has led us to establish operations in diverse markets, serve diverse patient communities and leverage diverse talents and ideas to champion innovation and health equality for patients. Diversity and inclusion, one of our six “PTC Expectations” has been…
    Our Culture
    May 19, 2023
    4 minutes
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  3. DuchenneCAN on a NASA mission with Ethan

    Ethan Higginbotham’s passion for astronomy and physics is “out of this world.” His interest in all things outer space influenced him to pursue an internship with NASA, and one day, he hopes to broaden his astrophysics knowledge through completing a physics PhD. Ethan, who describes himself as determined and driven, shows that people living with…
    Rare Disease Community
    May 18, 2023
    2 minutes
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  4. 25 Remarkable Stories: Tenacious Ferocity

    Jonathan Piacentino would tell you his proudest life moment was finally asking out a girl after working up the courage for many months. His mom, Christine, would disagree: “What about graduating college, isn’t that your proudest moment?” she asks him, laughing. Christine is personally very proud of her work with the Parent Project Muscular Dystrophy…
    Rare Disease Community
    May 17, 2023
    3 minutes
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  5. Why We Must Consider the ‘Totality of Evidence’ for Novel Rare Disease Therapies

    Christian Werner, Executive Director, Global Medical Affairs, Global DMD Lead, PTC, believes the “totality of evidence” provides an important understanding of the impact of a unique treatment. In this article, he shares strategies for broadening the adoption of the totality of evidence. As a physician working in rare disease drug development, I am convinced that…
    Science & Innovation
    May 15, 2023
    2 minutes
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  6. 25 Remarkable Stories: A Pioneering Advocate

    Kelly Heger became a nurse because of her daughter, Jillian. When Jillian was a baby, Kelly and her husband, Bruce, knew something was wrong. She had a very low temperature, and soon she began experiencing what is called an oculogyric crisis; Jillian’s eyes would roll upward and her neck would forcefully turn to one side…
    Rare Disease Community
    May 10, 2023
    2 minutes
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  7. Jenna Woodard: ‘The HD community has held my hand every step of the way…’

    Jenna Woodard is a Huntington’s disease (HD) patient and advocate from Albany, NY. She serves as an advocacy chair, youth mentor and ambassador for the Huntington’s Disease Youth Organization (HDYO). Do you have a rare disease story to share? Contact our team at shareyourstory@ptcbio.com. Hi, my name is Jenna. I am 22 years old, and…
    Rare Disease Community
    May 8, 2023
    2 minutes
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  8. EU Diversity Month 2023: Assessing Diversity and Inclusion at PTC

    This May, we are excited to celebrate EU Diversity Month and raise awareness about the importance of diversity and inclusion in the workplace and across society. Our Multicultural Alliance Group of Inclusive Communities (MAGIC) employee resource group (ERG) is conducting a number of activities this month that are aligned to this year’s theme, “assessing workplace…
    Our Culture
    May 5, 2023
    3 minutes
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  9. Committed to Doing our Part: 2022 Corporate Sustainability Report Highlights

    Last week, we were thrilled to publish the PTC 2022 Corporate Sustainability Report. “Committed to doing our part” is a comprehensive look at our science, support for rare disease patient communities and access to care, our strengths-based learning culture, global equality, diversity and inclusion, efforts to help the environment, and much more. As a growing…
    Community, Our Culture, Rare Disease Community, Responsibility
    May 3, 2023
    3 minutes
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  10. Take on Duchenne Podcast: A Mother and Carrier of Duchenne Muscular Dystrophy Shares Her Journey

    Elizabeth’s journey with Duchenne Muscular Dystrophy (DMD) began when her son was diagnosed at the age of five. In this episode, Elizabeth reflects on the diagnosis of her son, her daughter’s and her own carrier diagnosis, family life, her coping mechanisms and management of the disease. Although her and her family’s lives have been impacted…
    Rare Disease Community
    May 1, 2023
    2 minutes
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